International Plasma and Fractionation Association, Amsterdam, The Netherlands.
Faculty of Medicine and Health Sciences, The University of Western Australia (M509), Perth, Australia.
Vox Sang. 2024 Sep;119(9):1012-1020. doi: 10.1111/vox.13698. Epub 2024 Jun 26.
Plasma-derived medicinal products (PDMPs) are recognized internationally as essential medicines required to treat various acute and chronic conditions including congenital deficiencies of plasma proteins in haemophilia and primary immune deficiency. Global provision of these medicines is dominated by a small number of commercial companies, influencing the price and availability of the products. Achieving a level of strategic independence from this dominance is now seen as a public health priority in many countries. During the Regional Congress of the International Society for Blood Transfusion (ISBT) in Cape Town, South Africa, in November 2023, around 50 delegates from 24 countries participated in a workshop (WS) organized jointly by the International Plasma and Fractionation Association (IPFA) and the ISBT Working Party on Global Blood Safety on pathways towards provision of PDMPs from domestic plasma independent of commercial purchase in the open market. The WS was structured around three themes, each addressed by a separate group: Quality/safety requirements for plasma for fractionation (PfF) Stepwise access for safe plasma proteins Approaching contract fractionation A synthesis of conclusions from these groups included the following: The need to acquire support from government authorities for a national plasma policy, recognizing the difficulties posed by unstable political and bureaucratic environments. The value of embedding plasma and PDMPs within a patient blood management (PBM) paradigm to promote optimal clinical use of PDMPs. Training of blood/plasma collection personnel in the relevant principles of Good Manufacturing Practice (GMP), coupled with regulatory oversight of plasma product production in the engaged jurisdictions. Appreciation that limited access to contract fractionation may necessitate a stepwise approach, which may include small-scale preparation of versions of essential plasma proteins as an intermediate phase towards the manufacture of industrial-scale PDMPs from domestic plasma.
血浆衍生药品（PDMPs）在国际上被公认为是治疗各种急性和慢性疾病的基本药物，包括血友病和原发性免疫缺陷症等血浆蛋白先天性缺乏症。这些药物的全球供应主要由少数商业公司主导，这影响了产品的价格和供应。在许多国家，实现从这种主导地位获得战略独立，现在被视为公共卫生的优先事项。在 2023 年 11 月于南非开普敦举行的国际输血协会（ISBT）区域大会期间，来自 24 个国家的约 50 名代表参加了由国际血浆和分离协会（IPFA）和 ISBT 全球血液安全工作组联合组织的一个研讨会（WS），该研讨会探讨了在不依赖公开市场商业采购的情况下，从国内血浆中提供 PDMP 的途径。该 WS 围绕三个主题展开，每个主题由一个单独的小组负责：用于分离的血浆的质量/安全性要求逐步获得安全的血浆蛋白接近合同分离对这些小组的结论进行综合包括以下内容：需要从政府当局获得国家血浆政策的支持，认识到不稳定的政治和官僚环境带来的困难。将血浆和 PDMP 嵌入患者血液管理（PBM）范式中的价值，以促进 PDMP 的最佳临床使用。对血液/血浆采集人员进行良好生产规范（GMP）相关原则的培训，并在参与司法管辖区对血浆产品生产进行监管监督。认识到有限的合同分离机会可能需要逐步进行，其中可能包括小规模制备基本血浆蛋白的版本，作为从国内血浆制造工业规模 PDMP 的中间阶段。
